MFT selected as first UK site to roll out life-saving gene therapy for rare disease

Manchester University NHS Foundation Trust (MFT) has been selected as the first UK site for an NHS roll-out of a new life-saving gene therapy for babies affected by a rare disease.

The gene therapy – known by its brand name Libmeldy – is used to treat metachromatic leukodystrophy (MLD), which ’causes severe damage to a child’s nervous system and organs’, and ‘results in a life expectancy of between five and eight years’.

The treatment will be delivered by a specialist service through Royal Manchester Children’s Hospital, in collaboration with Manchester’s centre for Genomic Medicine at Saint Mary’s Hospital.

The trust will become the only site in the UK to administer the treatment, and just one of five in all of Europe.

With a reported list price of £2.8 million, the therapy is believed to be the most expensive drug in the world, but will be offered to young patients on the NHS after the health service negotiated a confidential discount.

Amanda Pritchard, Chief Executive, NHS said: “This revolutionary drug is a life-saver for the babies and young children who suffer from this devastating hereditary disorder and will spare their families untold heartache and grief.

“The deal we have struck is just the latest example of NHS England using its commercial capabilities to make good on the NHS Long Term Plan commitment to provide patients with cutting-edge treatments and therapies at a price that is fair to taxpayers.

“It also shows that while rolling out the world-leading NHS COVID-19 vaccination programme, and caring for people with the virus, the health service is also doing its very best to care for millions of patients with other conditions.”

Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at MFT and Clinical Director of NIHR Manchester Clinical Research Facility at Royal Manchester Children’s Hospital, added: “This is enormously welcome news for all families affected by MLD, a progressive, life-limiting condition for which there were previously no approved treatment options. While there are various sub-types of the condition, in its later stages, all forms largely result in children losing their ability to move and speak.”